The Hedgehog (Hh) signaling pathway is important in animal development, and has been highly conserved throughout evolution. Moreover, aberrant Hh signaling has been associated with the development and growth of many human cancers. As a result, the Hh signaling pathway has been a target in the research and development efforts of biotech and pharmaceutical companies to develop novel, non-toxic cancer treatments. Despite the massive scale of the resources dedicated to this approach, however, a Hh pathway antagonist that can be tested clinically for use in cancer therapy has not been achieved. Thus, there remains a need for the identification and development of drugs that act as Hh pathway antagonists.
First, identifying established drugs that also act as Hh pathway antagonists, and then using their already established toxicity profiles would facilitate the identification of Hh antagonists, and would more rapidly bridge the gap between the lab and the clinic. Thus, the present invention fills the need in the art for Hh pathway antagonists by providing identification of drugs with already established toxicity and pharmacokinetic profiles that can be used as compounds to inhibit Hh pathway activity, and treat a disorder or disease associated with aberrant Hh signaling, such as cancer.